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MDA Funds Expansion of Facioscapulohumeral Muscular Dystrophy Clinical Research Network

$1.2 million investment will support seven medical centers that specialize in FSHD research and clinical care

CHICAGO, June 20, 2018 – The Muscular Dystrophy Association announces the award of an MDA clinical research network grant totaling $1.2 million over three years to develop and maintain a core facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (CTRN). The goal of the research network is to spur advances in FSHD research and expedite the development of new therapies for FSHD.

Recent breakthroughs in FSHD research have identified the underlying molecular cause of the disease, and a number of drug companies currently are working to develop and test targeted therapeutic strategies to treat it. Consequently, there is an urgent need to establish the tools necessary to execute high-quality clinical trials in FSHD.

“MDA is pleased to support the expansion of this critical infrastructure for facioscapulohumeral muscular dystrophy research and therapy development,” said MDA Scientific Program Officer Laura Hagerty, Ph.D. “Today is FSHD Awareness Day, and it’s exciting to see and share with the FSHD community the progress taking place, with a rapidly growing pipeline of FSHD drugs in development.

“In order to keep that pipeline moving, however, it’s critical to ensure that clinical trials can be conducted quickly and efficiently to test these potential new treatments,” Hagerty added. “MDA support will enable the FSHD Clinical Trial Research Network to build a team of trained clinical evaluators all working with standardized approaches and develop regulatory strategies optimized to streamline drug trials.”

Successful clinical trials depend on several factors, including the ability to recruit patients, patient access, a precise understanding of the natural history of FSHD and the major contributors its variability, and reliable outcome measures that are sensitive to change in FSHD.

“Meetings with industry, advocacy groups and FSHD researchers have identified several gaps in our clinical trial arsenal and have pinpointed clinical trial planning as a major goal for the community,” said principal investigator Jeffrey Statland, M.D. “Not only will the FSHD CTRN help close gaps in trial readiness, but a network of sites will be created with a centralized streamlined regulatory process, specific common expertise in FSHD and an engaged patient population ready to conduct efficient, high-quality clinical trials.”

The network comprises seven medical centers with significant expertise in FSHD research and clinical care. Centers include:

  • California: University of California Los Angeles — Perry Shieh, M.D., Ph.D., site principal investigator
  • Kansas: University of Kansas Medical Center — Jeffrey Statland, M.D., site principal investigator
  • Maryland: Kennedy Krieger Institute — Kathryn Wagner, M.D., site principal investigator
  • New York: University of Rochester Medical Center — Rabi Tawil, M.D., site principal investigator
  • Ohio: The Ohio State University — John T. Kissel, M.D., site principal investigator
  • Utah: University of Utah — Nicholas Johnson, M.D., site principal investigator
  • Washington: University of Washington — Leo H. Wang, M.D., Ph.D., site principal investigator

MDA has funded more than $22 million in FSHD research since 1950, and including this most recent award, is currently is funding 10 active FSHD grants (with another two pending), with a total funding commitment of more than $3 million.

The new grant was approved by MDA’s Board of Directors after careful deliberations and analysis by MDA advisors and research staff. Currently, MDA is funding 177 research projects around the world, with another 28 pending.

About the Muscular Dystrophy Association
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at

Mary Fiance
Director, Public Relations and Communications
Muscular Dystrophy Association
646-992-2908 c: 917.538.5895